Two CRISPR-based biotech companies, one in Europe and one in the US, aim to start clinical trials this year, the Financial Times reports.
The Switzerland-based CRISPR Therapeutics has applied to regulators in Europe to seek approval to test its treatment for beta thalassemia, while the US-based Editas Medicine plans to soon ask the US Food and Drug Administration for the go-ahead to test its treatment for a type of congenital blindness, FT says. It adds that CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics hope to apply their approaches to a suite of diseases, including cancer, cystic fibrosis, hemophilia and Duchenne muscular dystrophy.
Diseases for which there are few available treatments will be the first to be tackled by gene-editing approaches, Katrine Bosley, chief executive of Editas, tells FT, as questions of safety and risk are still being addressed.
Clinicians in China have also been moving ahead with human studies of CRISPR-based treatments. For instance, Hangzhou Cancer Hospital researchers are using CRISPR to knock out the PD-1 gene within cancer patients' T cells to ramp up their immune response to tumor cells. Meanwhile, University of Pennsylvania researchers are about to begin their own trial editing the PD-1 and TCR genes within the T cells of patients with myeloma, melanoma, or sarcoma.